Bylvay granted orphan drug designation for Alagille syndrome

Will compete with Livmarli

MFDS announces designation effective October 1… Central Pharmaceutical Affairs Council recognizes the rationale for granting designation

Livmarli in final reimbursement talks, expected to improve patient access

From this month, Bylvay Cap (odevixibat, Ipsen Korea)—which was recently listed for reimbursement as a treatment for progressive familial intrahepatic cholestasis (PFIC)—has been designated as an orphan drug for treating cholestasis-associated symptoms in patients with Alagille syndrome.

 

The orphan designation is expected to accelerate the indication expansion, as it provides benefits such as expedited review and exemption from application fees.

 

As a result, Bylvay is now poised to compete with GC Biopharma’s Livmarli oral solution (maralixibat chloride), which is currently in the final stage of pricing negotiations for reimbursement for Alagille syndrome in Korea.

 

According to industry sources on the 15th, the Ministry of Food and Drug Safety announced the designation of Bylvay Cap as an orphan drug for treating Alagille syndrome, effective as of the 1st of this month.

 

Bylvay Cap obtained domestic marketing authorization last August as a treatment for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) aged three months or older.

 

This month, it also succeeded in securing reimbursement listing for the same indication.

 

This drug was selected for the parallel approval-evaluation-negotiation pilot program, enabling it to shorten the time to approval and reimbursement listing compared to other drugs.

 

Its recent designation as an orphan drug for Alagille syndrome is expected to further expedite the process for adding this new indication.

 

Orphan drug designation grants access to expedited review and waives application fees.

 

Additionally, even before final approval, the drug can be supplied to patients through the Korea Orphan & Essential Drug Center.

 

Currently, the only approved treatment for Alagille syndrome in Korea is GC Biopharma’s Livmarli oral solution, which was approved in February 2023 for the treatment of cholestatic pruritus in patients aged three months and older with Alagille syndrome (ALGS).

 

Livmarli is also designated as an orphan drug in Korea.

 

In August, Livmarli passed review by the Health Insurance Review and Assessment Service’s (HIRA) Drug Reimbursement Evaluation Committee and is now in final pricing negotiations with the National Health Insurance Service (NHIS).

 

Once listed for reimbursement, Alagille syndrome patients will be able to use Livmarli with insurance benefits in Korea.

 

Alagille syndrome is a condition where the number of bile ducts within the liver is significantly reduced, preventing bile from being excreted from the liver and causing it to accumulate and cause complications in the cardiovascular, skeletal, ocular, and dermatologic systems.

 

The disease primarily affects children, and due to the lack of effective treatments, it causes significant suffering for patients and their families.

 

There are currently an estimated 136 patients with the condition in Korea.

 

During its review, the Central Pharmaceutical Affairs Council deemed the orphan drug designation for Bylvay to be appropriate, considering the drug’s safety, efficacy, and supply feasibility.

 

The fact that Bylvay has also been designated as an orphan drug for Alagille syndrome in the United States and Europe was also taken into account.

 

As such, once Bylvay obtains formal approval for the Alagille syndrome indication, it is expected to directly compete with GC Biopharma’s Livmarli, which is nearing reimbursement approval.

 

Furthermore, the orphan drug designation is anticipated to somewhat improve patient access.